Core Insights - Maze Therapeutics, Inc. has shared promising clinical results for MZE782, an oral small molecule targeting the solute transporter SLC6A19, which may serve as a best-in-class therapy for phenylketonuria (PKU) and a first-in-class therapy for chronic kidney disease (CKD) [1] Pharmacokinetics (PK) - MZE782 exhibited a favorable plasma pharmacokinetic profile with consistent absorption, a tmax of six hours, and a half-life of 11 hours [2] - Exposure to MZE782 increased proportionally with dose, achieving steady-state by Day 3, indicating potential for once- or twice-daily dosing in Phase 2 trials [2] Neutral Amino Acid Excretion - MZE782 resulted in dose-dependent increases in 24-hour urinary excretion of neutral amino acids Phe and Gln, confirming target engagement and SLC6A19 inhibition [3] Urinary Excretion Results - A 39-fold increase in urinary Phe excretion was observed with a single 960 mg dose, and a 42-fold increase was noted on Day 7 in the 240 mg twice-daily cohort [4] - A 55-fold increase in urinary Gln excretion was recorded with a single 960 mg dose, and a 68-fold increase was observed on Day 7 in the 240 mg twice-daily cohort [4] Estimated Glomerular Filtration Rate (eGFR) - MZE782 demonstrated a dose-dependent initial dip in eGFR over seven days of dosing, comparable to effects seen in patients starting SGLT2 and RAAS inhibitors [5] Next Steps - Maze plans to initiate two Phase 2 proof-of-concept trials for MZE782 in 2026, focusing on plasma Phe reduction in PKU and proteinuria reduction in CKD [6] - MZE782 was well tolerated across all doses, with no serious or treatment-related adverse events leading to discontinuation [6] Financial Update - Maze Therapeutics announced an oversubscribed private placement of approximately $150 million, consisting of 4 million shares at $16.25 per share [7] - Following the announcement, Maze Therapeutics shares rose by 52.15% to $24.37, reaching a new 52-week high [7]